LCA (Leber Congenital Amaurosis), RP (Retinitis Pigmentosa)
Conditions
Brief summary
The purpose of this study is: * to evaluate the safety of oral QLT091001 * to evaluate whether 7-day treatment with oral QLT091001 can improve visual function in subjects with LCA or RP due to RPE65 or LRAT mutations * to evaluate duration of visual function improvement (if observed)
Interventions
DRUGQLT091001
oral QLT091001 administered once daily for 7 days
Sponsors
QLT Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
5 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
* Subjects diagnosed with LCA or RP (with a mutation in either RPE65 or LRAT) * Subjects with LCA must be 5-65 years of age * Subjects with RP must be 18-65 years of age * Subjects who have a best-corrected visual acuity of 3 letters or better (20/800 Snellen equivalent) or viable photoreceptors on OCT/FAF.
Exclusion criteria
* Subjects who are actively participating in an experimental therapy study or who have received experimental therapy within 60 days of Day 0. * Subjects with any clinically important abnormal physical finding at Screening. * Subjects who have taken any prescription or investigational oral retinoid medication (e.g., Accutane,® Soriatane®) within 6 months of Day 0 and subjects who did not tolerate their previous oral retinoid medication will be excluded regardless of the time of last exposure. * Subjects with a history of diabetes or chronic hyperlipidemia, hepatitis, pancreatitis, or cirrhosis. * Subjects who have taken any supplements containing ≥10,000 IU vitamin A within 60 days of screening
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Visual Field | 12 months |
Secondary
| Measure | Time frame |
|---|---|
| Vital signs, clinical laboratory tests, electrocardiogram (ECG), and adverse events | 12 months |
Countries
Canada, Germany, Netherlands, United Kingdom, United States
Outcome results
None listed